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Levofloxacin prophylaxis in pediatric oncology and hematopoietic stem cell transplantation: a literature review. 小儿肿瘤学和造血干细胞移植中的左氧氟沙星预防疗法:文献综述。
IF 1.2 4区 医学 Q4 HEMATOLOGY Pub Date : 2024-09-01 Epub Date: 2024-07-08 DOI: 10.1080/08880018.2024.2353888
Jane Koo, Jeffrey Hord, Craig Gilliam, Mary Lynn Rae, Katherine Staubach, Katherine Nowacki, Anne Lyren, Maitreya Coffey, Christopher E Dandoy

Bloodstream infections (BSI) are one of the leading causes of morbidity and mortality in children and young adults receiving chemotherapy for malignancy or undergoing hematopoietic stem cell transplantation (HSCT). Antibiotic prophylaxis is commonly used to decrease the risk of BSI; however, antibiotics carry an inherent risk of complications. The aim of this manuscript is to review levofloxacin prophylaxis in pediatric oncology patients and HSCT recipients. We reviewed published literature on levofloxacin prophylaxis to prevent BSI in pediatric oncology patients and HSCT recipients. Nine manuscripts were identified. The use of levofloxacin is indicated in neutropenic children and young adults receiving intensive chemotherapy for leukemia or undergoing HSCT. These results support the efficacy of levofloxacin in pediatric patients with leukemia receiving intensive chemotherapy and should be considered in pediatric patients undergoing HSCT prior to engraftment.

在接受恶性肿瘤化疗或造血干细胞移植(HSCT)的儿童和年轻人中,血流感染(BSI)是导致发病和死亡的主要原因之一。抗生素预防是降低BSI风险的常用方法,但抗生素本身也存在并发症风险。本稿件旨在回顾儿童肿瘤患者和造血干细胞移植受者使用左氧氟沙星预防的情况。我们回顾了已发表的有关左氧氟沙星预防小儿肿瘤患者和造血干细胞移植受者 BSI 的文献。共找到九篇手稿。左氧氟沙星适用于接受白血病强化化疗或造血干细胞移植的中性粒细胞减少的儿童和年轻成人。这些结果支持左氧氟沙星在接受强化化疗的儿童白血病患者中的疗效,在接受造血干细胞移植的儿童患者中,也应考虑在移植前使用左氧氟沙星。
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引用次数: 0
Oncological pediatric early warning score: a dedicated tool to predict patient's clinical deterioration and need for pediatric intensive care treatment. 肿瘤儿科预警评分:预测患者临床病情恶化和儿科重症监护治疗需求的专用工具。
IF 1.2 4区 医学 Q4 HEMATOLOGY Pub Date : 2024-09-01 Epub Date: 2024-07-08 DOI: 10.1080/08880018.2024.2355543
Teresa Maccarana, Marta Pillon, Veronica Bertozzi, Elisa Carraro, Elena Cavallaro, Claudia Maria Bonardi, Luca Marchetto, Giulia Reggiani, Annalisa Tondo, Camilla Rosa, Rosanna Irene Comoretto, Angela Amigoni, Alessandra Biffi

Pediatric oncohematological patients frequently require PICU admission during their clinical history. The O-PEWS is a specific score developed to predict the need for PICU admission of oncohematological children. This study aimed at i) describing the trend of the O-PEWS in a cohort of patients hospitalized in the Pediatric Oncohematology ward and transferred to the PICU of Padua University Hospital, measured at different time-points in the 24 hours before PICU admission and to evaluate its association with mortality and presence of organ failure; ii) investigating the association between the recorded O-PEWS, and PIM3, number of organ failure and the need for ventilation, dialysis and inotropes.

This retrospective single-center study enrolled oncohematological children admitted to the PICU between 2017 and 2021. The O-PEWS, ranging between 0 and 15, was calculated on the available medical records and the TIPNet-Network database at 24 (T-24), 12 (T-12), 6 (T-6) and 0 (T0) hours before PICU admission.

RESULTS: 101 PICU admissions, related to 80 children, were registered. During the 24 hours prior to PICU admission, the O-PEWS progressively increased in all the patients. At T-24 the median O-PEWS was 3 (IQR 1-5), increasing to a median value of 6 (IQR 4-8) at T0. The O-PEWS was positively associated with mortality, organ failure and the need for ventilation at all the analyzed time-points and with the need for dialysis at T-6.

The O-PEWS appears as a useful tool for predicting early clinical deterioration in oncohematological patients and for anticipating the initiation of life-support treatments.

儿科血液病患者在临床病史中经常需要入住 PICU。O-PEWS是为预测儿童肿瘤患者是否需要入住PICU而开发的一种特殊评分。本研究旨在 i) 描述在帕多瓦大学医院儿科肿瘤病房住院并转入 PICU 的一组患者的 O-PEWS 变化趋势(在 PICU 入院前 24 小时内的不同时间点测量),并评估其与死亡率和器官衰竭的关联性;ii) 调查记录的 O-PEWS 与 PIM3、器官衰竭数量以及通气、透析和肌注需求之间的关联性。这项回顾性单中心研究纳入了2017年至2021年间入住PICU的患儿。根据现有病历和 TIPNet-Network 数据库计算了 PICU 入院前 24 小时(T-24)、12 小时(T-12)、6 小时(T-6)和 0 小时(T0)的 O-PEWS,范围在 0 至 15 之间。在进入 PICU 之前的 24 小时内,所有患者的 O-PEWS 都在逐渐增加。在 24 小时内,O-PEWS 的中位数为 3(IQR 1-5),在 24 小时后的第 0 小时,中位数增至 6(IQR 4-8)。在所有分析的时间点上,O-PEWS 都与死亡率、器官衰竭和通气需求呈正相关,在 T-6 时与透析需求呈正相关。
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引用次数: 0
Real-world implementation of North American and SIOP strategies for the treatment of Wilms tumor in Uruguay. 在乌拉圭实际实施北美和 SIOP 战略治疗 Wilms 肿瘤。
IF 1.2 4区 医学 Q4 HEMATOLOGY Pub Date : 2024-09-01 Epub Date: 2024-07-08 DOI: 10.1080/08880018.2024.2345662
Diana Vargas, Luisa Chantada, Bruno Cuturi, Anaulina Silveira, Ángeles Rodríguez, Luján Guerrero, Lucia Díaz, Mariela Castiglioni, Fabiana Morosini, Carolina Pages, Elizabeth Simón, Luis Castillo

Wilms tumor has been selected as an index tumor by the WHO Global Initiative for Childhood Cancer with the aim to improve cure rates worldwide. Nevertheless, there is a scarcity of published data on outcomes beyond those of the major cooperative groups. Therefore, we conducted a retrospective analysis including all patients with Wilms tumor treated at our referral center in Uruguay between 1995 and 2020. Treatment consisted of North American (NA) strategies in 23 cases (1995-2004), followed by the SIOP strategy in 35 cases thereafter. Staging included: I-II = 28, III = 7, IV = 14, and V = 9. There were no major surgical or medical complications; however, a delay in the administration of local radiotherapy was observed (median of 21 days after surgery). There were no cases of toxicity- or surgery-related deaths or treatment abandonment. Five-year probability of overall survival was 0.72 and 0.92 for the NA and SIOP groups, respectively. We conclude that outcomes were better for the SIOP strategy with no unexpected toxicities and high treatment compliance in both strategies. Timely implementation of radiotherapy was challenging.

世界卫生组织儿童癌症全球倡议将 Wilms 肿瘤选为指数肿瘤,旨在提高全球治愈率。然而,除了主要合作组的数据外,关于治疗效果的公开数据还很少。因此,我们对 1995 年至 2020 年期间在乌拉圭转诊中心接受治疗的所有 Wilms 肿瘤患者进行了回顾性分析。23例患者(1995-2004年)的治疗采用北美(NA)策略,35例采用SIOP策略。分期包括I-II期=28例,III期=7例,IV期=14例,V期=9例。手术或内科并无重大并发症,但局部放疗的实施出现了延迟(术后中位数为 21 天)。无中毒或手术相关死亡或放弃治疗的病例。NA组和SIOP组的五年总生存概率分别为0.72和0.92。我们的结论是,SIOP策略的疗效更好,两种策略均无意外毒性反应,治疗依从性高。及时实施放疗具有挑战性。
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引用次数: 0
Adjuvant immune checkpoint inhibitor therapy may benefit pediatric patients with stage III melanoma and sentinel lymph node positivity: a case series. 辅助免疫检查点抑制剂疗法可使Ⅲ期黑色素瘤和前哨淋巴结阳性的儿科患者获益:一个病例系列。
IF 1.2 4区 医学 Q4 HEMATOLOGY Pub Date : 2024-09-01 Epub Date: 2024-05-07 DOI: 10.1080/08880018.2024.2350455
Acacia Bowden, Jeanette Zambito, Jinia El-Feghaly, Jeffrey R Andolina

Melanoma is the most common skin cancer in children. While the current literature establishes treatment protocols for adult-type melanoma, very few pediatric-specific studies exist, and children are often excluded from melanoma clinical trials2. We report a case series of 23 pediatric patients aged 2-20 years old diagnosed with melanoma at the University of Rochester Medical Center between 1/1/2011 and 1/1/2022. 9/23 patients were Stage III; all patients underwent wide local excision and 9 received adjuvant therapies. 2/23 (8.7%) patients had recurrence of their malignancy after therapy while 21/23 (91.3%) remained without disease progression; 1 patient died from unknown cause, but the rest are alive and currently without disease. All patients whose initial therapy included nivolumab in addition to wide local excision did not have recurrence or progression of their disease. This case series highlights trends in the presentation, treatment, and outcomes of pediatric melanoma; however, additional multi-center studies are needed to establish the clinical utility of such features in pediatric melanoma.

黑色素瘤是儿童最常见的皮肤癌。目前的文献为成人型黑色素瘤制定了治疗方案,但针对儿童的研究却很少,而且儿童往往被排除在黑色素瘤临床试验之外2。我们报告了 2011 年 1 月 1 日至 2022 年 1 月 1 日期间在罗切斯特大学医学中心确诊为黑色素瘤的 23 名 2-20 岁儿童患者的系列病例。9/23例患者为III期;所有患者均接受了广泛局部切除术,9例接受了辅助治疗。2/23(8.7%)名患者在治疗后恶性肿瘤复发,21/23(91.3%)名患者的病情没有进展;1名患者死因不明,但其余患者均健在且目前没有患病。除了广泛局部切除术外,所有初始治疗包括 nivolumab 的患者都没有复发或病情进展。本系列病例强调了小儿黑色素瘤的表现、治疗和预后趋势;然而,要确定这些特征在小儿黑色素瘤中的临床实用性,还需要进行更多的多中心研究。
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引用次数: 0
Characteristics and complications of acute promyelocytic leukemia in children: an analysis of a national database. 儿童急性早幼粒细胞白血病的特征和并发症:全国数据库分析。
IF 1.2 4区 医学 Q4 HEMATOLOGY Pub Date : 2024-09-01 Epub Date: 2024-07-08 DOI: 10.1080/08880018.2024.2352727
Ariella Barhen, Paul A Martinez, Prithvi Sendi, Balagangadhar R Totapally

Acute promyelocytic leukemia (APL) is an uncommon subtype of acute myelogenous leukemia (AML) that was previously one of the most fatal forms of acute leukemia. With advances in diagnosis and treatment, APL has become one of the most curable myeloid leukemias. The major reason for treatment failure in APL is early death after initiation of treatment. We performed a retrospective cross-sectional analysis of the Healthcare Cost and Utilization Project 2016 and 2019 Kids' Inpatient Database, with the diagnosis of APL or AML not in remission as defined by ICD-10-CM codes. We compared complications and outcomes associated with APL and AML (exclusive of APL) in hospitalized children in the U.S. and described yearly national incidence. The national incidence of APL was 2.2 cases per million children per year. Children with APL were more likely to have cardiopulmonary complications (OR 1.79; CI 1.20-2.67; p = 0.004), coagulation abnormalities or DIC (OR 7.75; CI 5.81-10.34; p < 0.001), pulmonary hemorrhage (OR 2.18; CI 1.49-3.17; p < 0.001), and intracranial hemorrhage (OR 10.82; CI 5.90-19.85; p < 0.001) and less likely to have infectious complications (OR 0.48; CI 0.34-0.67; p < 0.001) compared to children with AML. In-hospital mortality rates were similar in children with APL and AML (4.2% vs 2.6%; OR 1.62; CI 0.86-3.06; p = 0.13), while the median length of stay for children who died from APL was shorter compared to AML (2 (IQR: 1-7) versus 25 (IQR: 5-66) days; p < 0.05). Hemorrhagic complications occur more often, and infectious complications occur less often in hospitalized children with APL compared to AML.

急性早幼粒细胞白血病(APL)是急性髓性白血病(AML)中一种不常见的亚型,曾是急性白血病中最致命的形式之一。随着诊断和治疗的进步,APL 已成为最容易治愈的髓系白血病之一。APL 治疗失败的主要原因是患者在开始治疗后过早死亡。我们对医疗成本与利用项目 2016 年和 2019 年儿童住院病人数据库进行了回顾性横断面分析,诊断结果为 APL 或未缓解的 AML(根据 ICD-10-CM 编码定义)。我们比较了美国住院儿童 APL 和 AML(不包括 APL)的相关并发症和结果,并描述了每年的全国发病率。APL的全国发病率为每年每百万儿童中有2.2例。患 APL 的儿童更有可能出现心肺并发症(OR 1.79;CI 1.20-2.67;P = 0.004)、凝血异常或 DIC(OR 7.75;CI 5.81-10.34;P P P P = 0.13),而死于 APL 的儿童的中位住院时间比死于 AML 的儿童短(2(IQR:1-7)天对 25(IQR:5-66)天;P = 0.004)。
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引用次数: 0
The relationship between liver stiffness by two-dimensional shear wave elastography and iron overload status in transfusion-dependent patients. 通过二维剪切波弹性成像检测输血依赖型患者肝脏硬度与铁超负荷状态之间的关系。
IF 1.2 4区 医学 Q4 HEMATOLOGY Pub Date : 2024-09-01 Epub Date: 2024-07-09 DOI: 10.1080/08880018.2024.2353900
Pimporn Puttawibul, Supika Kritsaneepaiboon, Thirachit Chotsampancharoen, Polathep Vichitkunakorn

Increased liver stiffness (LS) can be result of increased liver iron concentration (LIC) which may not yet be reflected in the liver fibrotic status. The objective of our study was to examine relationship between hemochromatosis, LS, and serum ferritin level in transfusion-dependent patients. We recruited all 70 transfusion-dependent patients, whose median age was 15, referred for evaluating LIC status by magnetic resonance imaging (MRI) followed by two-dimensional ultrasonography shear wave elastography (2D-SWE). Thalassemia beta affected the majority of the patients. The optimal cut point for prediction of severe hemochromatosis using median SWE (kPa) and SWV (m/s) was ≥ 7.0 kPa and ≥ 1.54 m/s, respectively, with sensitivity of 0.76 (95% confidence interval [CI] 0.55, 0.91) and, specificity of 0.69 (95%CI 0.53, 0.82). When combing the optimal cut point of SWE (kPa) at ≥ 7.0 and serum ferritin ≥ 4123 ng/mL, the sensitivity increased to 0.84 (95%CI 0.64, 0.95) with specificity of 0.67 (95%CI 0.50, 0.80), positive predictive value (PPV) of 0.60 (95%CI 0.42, 0.76), and negative predictive value (NPV) of 0.88 (95%CI 0.71, 0.96). Simultaneous tests of 2D-SWE and serum ferritin for prediction of severe hemochromatosis showed the highest sensitivity of 84% (95%CI 0.64-0.95), as compared to 2D-SWE alone at 76% (95%CI 0.55, 0.91) or serum ferritin alone at 44% (95%CI 0.24-0.65). We recommend measuring both 2D-SWE and serum ferritin in short interval follow up patients. Adding 2D-SWE to management guideline will help in deciding for aggressive adjustment of iron chelating medication and increased awareness of patients having severe hemochromatosis.

肝硬变(LS)的增加可能是肝铁浓度(LIC)增加的结果,而肝铁浓度的增加可能尚未反映在肝纤维化状态中。我们的研究旨在探讨输血依赖型患者血色素沉着病、肝硬变和血清铁蛋白水平之间的关系。我们招募了所有 70 名输血依赖型患者(中位年龄为 15 岁),通过磁共振成像(MRI)和二维超声剪切波弹性成像(2D-SWE)评估 LIC 状态。大多数患者都患有地中海贫血。使用中位 SWE(kPa)和 SWV(m/s)预测重度血色素沉着病的最佳切点分别是≥7.0 kPa 和≥1.54 m/s,灵敏度为 0.76(95% 置信区间 [CI] 0.55,0.91),特异性为 0.69(95%CI 0.53,0.82)。将 SWE (kPa) ≥ 7.0 和血清铁蛋白 ≥ 4123 ng/mL 的最佳切点结合起来,灵敏度增加到 0.84(95%CI 0.64,0.95),特异性为 0.67(95%CI 0.50,0.80),阳性预测值 (PPV) 为 0.60(95%CI 0.42,0.76),阴性预测值 (NPV) 为 0.88(95%CI 0.71,0.96)。同时检测 2D-SWE 和血清铁蛋白预测重度血色病的灵敏度最高,为 84% (95%CI 0.64-0.95),而单独检测 2D-SWE 的灵敏度为 76% (95%CI 0.55, 0.91) 或单独检测血清铁蛋白的灵敏度为 44% (95%CI 0.24-0.65)。我们建议在短间隔随访患者中同时测量 2D-SWE 和血清铁蛋白。将 2D-SWE 纳入管理指南将有助于决定是否积极调整除铁药物,并提高对严重血色沉着病患者的认识。
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引用次数: 0
Myelofibrosis associated with immune cytopenia in an infant: a diagnostic and therapeutic challenge. 婴儿骨髓纤维化伴有免疫细胞减少症:诊断和治疗难题。
IF 1.2 4区 医学 Q4 HEMATOLOGY Pub Date : 2024-08-01 Epub Date: 2024-05-07 DOI: 10.1080/08880018.2024.2350424
Yessenia L Molina, Luis Blasco-Santana, Alejandro Sanz, Cristina Julia Blázquez Gómez, Josune Zubicaray, June Iriondo, Jesús González de Pablo, Julián Sevilla, Elena Sebastián
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引用次数: 0
The Italian Consensus Conference on the role of rehabilitation for children and adolescents with leukemia, central nervous system tumors, and bone cancer, part 2: general principles for the rehabilitation treatment of motor function impairments. 意大利关于白血病、中枢神经系统肿瘤和骨癌儿童和青少年康复作用的共识会议,第二部分:运动功能障碍康复治疗的一般原则。
IF 1.2 4区 医学 Q4 HEMATOLOGY Pub Date : 2024-08-01 Epub Date: 2024-07-10 DOI: 10.1080/08880018.2024.2353360
Francesca Rossi, Stefano Botti, Mattia Morri, Sebastian Asaftei, Daniele Bertin, Simona Breggiè, Roberto Casalaz, Marta Cervo, Paola Ciullini, Monica Coppo, Annalisa Cornelli, Maria Esposito, Miriana Ferrarese, Marina Ghetti, Lucia Longo, Gabriella Naretto, Nicoletta Orsini, Daniele Panzeri, Chiara Pellegrini, Michela Peranzoni, Antonella Perna, Nadine Petit, Fabiola Picone, Gianna Pittorru, Debora Raffa, Veronica Recchiuti, Lucia Rizzato, Marina Sarzana, Raffaella Sensi, Franca Fagioli, Federica Ricci

In Italy, 1400 children and 800 adolescents are diagnosed with cancer every year. About 80% of them can be cured but are at high risk of experiencing severe side effects, many of which respond to rehabilitation treatment. Due to the paucity of literature on this topic, the Italian Association of Pediatric Hematology and Oncology organized a Consensus Conference on the role of rehabilitation of motor impairments in children/adolescents affected by leukemia, central nervous system tumors, and bone cancer to state recommendations to improve clinical practice. This paper includes the consensus on the rehabilitation of children and adolescents with these cancers.

在意大利,每年有 1400 名儿童和 800 名青少年被诊断出患有癌症。其中约 80% 可以治愈,但出现严重副作用的风险很高,其中许多副作用对康复治疗有反应。由于这方面的文献很少,意大利小儿血液学和肿瘤学协会组织了一次共识会议,就白血病、中枢神经系统肿瘤和骨癌患儿/青少年运动障碍康复治疗的作用提出建议,以改进临床实践。本文件包括关于这些癌症儿童和青少年康复的共识。
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引用次数: 0
Management of a major varicella zoster exposure in a pediatric oncology population. 在儿科肿瘤患者中处理重大水痘带状疱疹暴露。
IF 1.2 4区 医学 Q4 HEMATOLOGY Pub Date : 2024-08-01 Epub Date: 2024-02-20 DOI: 10.1080/08880018.2024.2315456
Carol Rossetto, Kent Sepkowitz, Jill Ackerman, Rachel Corke, Nina J Pickett, Wini Cudjoe, Janet Eagan, Mini Kamboj, Richard J O'Reilly, Farid Boulad

Management of the exposure of pediatric oncology patients to varicella zoster virus (VZV) is controversial. We report the exposure of 56 patients to a single child with chicken pox at a pediatric cancer housing facility and describe our strategic approach for their management. We reviewed the immune and clinical status of 56 children with cancer receiving ongoing treatment at Memorial Sloan Kettering Cancer Center (MSK) who, while living at a pediatric cancer housing facility, were exposed to the index patient. The management of patients exposed included: (1) determination of immune status, (2) availability of vaccination history or VZV disease prophylaxis, (3) exposure status and subsequent isolation during the period of incubation, and (4) VZV disease prophylaxis. In addition to the 56 patients exposed to the index case, eight children with cancer treated at other facilities and 11 healthy siblings living in the facility were exposed. Of the 56 MSK patients, 21 were classified as immunosuppressed and received varicella zoster immune globulin (human), intravenous standard immune globulin, or acyclovir based on serostatus and immune function. The cohort was followed for 4 weeks after the exposure and no secondary infections were diagnosed. We performed a risk assessment and created a management plan to control and prevent further exposure and development of disease. No secondary cases developed. This strategic approach could serve as a model for the management of VZV exposure for other pediatric oncology centers.

儿科肿瘤患者暴露于水痘带状疱疹病毒(VZV)的处理方法存在争议。我们报告了一家儿科癌症住院机构的 56 名患者与一名水痘患儿的接触情况,并介绍了我们的管理策略。我们回顾了在纪念斯隆-凯特琳癌症中心(MSK)接受持续治疗的 56 名癌症患儿的免疫和临床状况,这些患儿住在儿科癌症病房设施时曾接触过指标病人。对暴露患者的管理包括(1) 确定免疫状态,(2) 提供疫苗接种史或 VZV 疾病预防措施,(3) 暴露状态和随后的潜伏期隔离,以及 (4) VZV 疾病预防措施。除了 56 名暴露于该病例的患者外,还有 8 名在其他机构接受治疗的癌症患儿和 11 名居住在该机构的健康兄弟姐妹也受到了感染。在 56 名 MSK 患者中,21 人被列为免疫抑制患者,并根据血清状态和免疫功能接受了水痘带状疱疹免疫球蛋白(人)、静脉注射标准免疫球蛋白或阿昔洛韦治疗。接触后,我们对这些患者进行了为期 4 周的随访,没有发现继发性感染。我们进行了风险评估并制定了管理计划,以控制和预防进一步的接触和疾病发展。没有出现继发病例。这一战略方法可作为其他儿科肿瘤中心管理 VZV 暴露的典范。
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引用次数: 0
Co-transplantation of umbilical cord mesenchymal stem cells and peripheral blood stem cells in children and adolescents with refractory or relapsed severe aplastic anemia. 为难治性或复发性重型再生障碍性贫血的儿童和青少年联合移植脐带间充质干细胞和外周血干细胞。
IF 1.2 4区 医学 Q4 HEMATOLOGY Pub Date : 2024-08-01 Epub Date: 2024-03-04 DOI: 10.1080/08880018.2024.2324394
Fangfang Yuan, Minghui Li, Xudong Wei, Yuewen Fu

To evaluate the co-transplantation efficacy of umbilical cord mesenchymal stem cells (UC-MSCs) and peripheral blood stem cells (PBSCs) as a novel approach for refractory or relapsed severe aplastic anemia (R/R SAA) in children and adolescents, thirty-two children and adolescents diagnosed with R/R SAA underwent a retrospective chart review. The patients were categorized into two groups based on the source of PBSCs: the matched sibling donor (MSD) group and the unrelated donor (UD) group. No adverse events related to UC-MSC infusion occurred in any of the patients. The median time for neutrophil engraftment was 13 days (range: 10-23 days), and for platelets, it was 15 days (range: 11-28 days). Acute GVHD of Grade I-II and moderate chronic GVHD were observed in 21.8 and 12.5% of cases, respectively. No statistically significant differences were found between the MSD and UD groups in terms of engraftment, GVHD, and complications, including infection and hemorrhagic cystitis. The median follow-up time was 38.6 months (range: 1.4-140.8 months). As of October 31, 2021, five patients had succumbed, while 27 (84.4%) survived. The 5-year OS rate showed no statistically significant difference between the MSD and UD groups (84.8 ± 10.0 vs. 82.4 ± 9.2%, p = 0.674). In conclusion, the application of UC-MSCs in the treatment of R/R SAA in PBSC transplantation is reliable and safe, they had no graft rejection, low incidence of severe GVHD which may have been contributed by the co-infusion of MSC.

为了评估脐带间充质干细胞(UC-MSCs)和外周血干细胞(PBSCs)联合移植作为治疗儿童和青少年难治性或复发性重型再生障碍性贫血(R/R SAA)新方法的疗效,我们对32名确诊为R/R SAA的儿童和青少年进行了回顾性病历审查。根据PBSCs的来源将患者分为两组:匹配的兄弟姐妹供体(MSD)组和非亲属供体(UD)组。所有患者均未发生与 UC-MSC 输注相关的不良事件。中性粒细胞移植的中位时间为13天(范围:10-23天),血小板移植的中位时间为15天(范围:11-28天)。分别有21.8%和12.5%的病例观察到I-II级急性GVHD和中度慢性GVHD。MSD组和UD组在移植、GVHD和并发症(包括感染和出血性膀胱炎)方面没有明显的统计学差异。中位随访时间为 38.6 个月(范围:1.4-140.8 个月)。截至 2021 年 10 月 31 日,5 名患者死亡,27 名患者(84.4%)存活。MSD组和UD组的5年生存率无明显统计学差异(84.8 ± 10.0 vs. 82.4 ± 9.2%,P = 0.674)。总之,应用 UC 间充质干细胞治疗 R/R SAA 的 PBSC 移植是可靠和安全的,他们没有发生移植物排斥反应,严重 GVHD 的发生率也很低,这可能与间充质干细胞的联合灌注有关。
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引用次数: 0
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Pediatric Hematology and Oncology
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